Therapeutic/Technology: Therapeutic Approach
Combating COVID-19 using human PSC-derived NK cells
Research Objective We propose to generate NK cells with enhanced immunity from gene-edited human PSCs and use the resultant NK cells to kill SARS-CoV-2-infected cells to combat against COVID-19. Impact The use of gene-edited hPSCs as a source for genetically engineered NK cells will allow us to generate effective immunotherapy for COVID-19 that has no […]
Development of COVID-19 Antiviral Therapy Using Human iPSC-Derived Lung Organoids
Research Objective To develop a new therapy for COVID-19 using human iPSC-derived lung organoids that targets SARS-CoV-2 protease known as the virus’ “Achilles Heel” Impact Our work, if successful, will bring a class of new drugs directly targeting viral enzyme and open the door for future COVID therapies. Major Proposed Activities Complete synthesis and testing […]
Human-induced pluripotent stem cell-derived glial enriched progenitors to treat white matter stroke and vascular dementia.
Research Objective This grant proposes development of a stem cell based therapy that is derived from human induced pluripotent stem cells. These cells are in the form of a brain support cell, an astrocyte. Impact The cell candidate will treat vascular dementia, the second leading cause of dementia, and stroke by overcoming a bottleneck in […]
Optimization of a human interneuron cell therapy for traumatic brain injury
Research Objective A cell therapy product comprised of inhibitory neurons that can migrate, integrate and restore neurologic function after traumatic brain injury. Impact Traumatic brain injury Major Proposed Activities Examine the most effective dose and safety profile of human iPSC-derived MGE cells grafted into rodent hippocampus. Determine whether human iPSC-derived MGE cells mature into appropriate […]
Development of a SYF2 antisense oligonucleotide (ASO) treatment for ALS
Research Objective We will develop an antisense oligonucleotide, or DNA therapy for diverse forms of amyotrophic lateral sclerosis (ALS). Impact ALS is fatal and incurable, and if successful, we will develop a treatment that slows or stops ALS progression across a broad range of patients. Major Proposed Activities Selection of the lead drug by testing […]
Transplantation of Pluripotent Stem Cell Derived Microglia for the Treatment of Adult-onset Leukoencephalopathy (HDLS/ALSP)
Research Objective We propose to investigate the transplantation of pluripotent stem cell derived microglia as a potential therapy for the devastating neurological disease; Adult-onset leukoencephalopathy (ALSP/HDLS). Impact The most immediately impacted condition will be ALSP. However, further examination of the safety of human microglial transplantation will have broad implications for many neurodegenerative disorders Major Proposed […]
The First Orally Delivered Cell Therapy for the Treatment of Inflammatory Bowel Disease
Research Objective The goal of this project is to develop the first ORAL cell therapy as a breakthrough treatment for inflammatory bowel disease [IBD]. Impact We engineered a new way to deliver cells ORALLY instead of by injection. In doing so, we will better reach the inflamed tissues and provide a much-needed new treatment for […]
Dual angiogenic and immunomodulating nanotechnology for subcutaneous stem cell derived islet transplantation for the treatment of diabetes
Research Objective Functional human islet like organoids differentiated from human pluripotent stem cells. Impact Providing the immediate cell therapeutic candidate for clinical trial of diabetic patients. Major Proposed Activities Fabrication and characterization of the injectable immunomodulating and pro-angiogenic material components: HA hydrogel, heparin nanoparticles and VEGF clusters. Generation of human islet like organoids from pluripotent […]
Hematopoietic Stem Cell Gene Therapy for X-linked Agammaglobulinemia
Research Objective The objectives of this study are to advance a stem cell gene therapy for the immunodeficiency XLA, defining the final therapeutic candidate and showing therapeutic activity in a relevant mouse model. Impact XLA can be treated with chronic immunoglobulin replacement, but may be sub-optimal due to infections and inflammatory complications. Stem cell gene […]
Human iPSC-derived chimeric antigen receptor-expressing macrophages for cancer treatment
Research Objective These studies will produce a new CAR-targeted iPSC-derived macrophage-based cell therapy product for treatment of refractory malignancies such as ovarian cancer. Impact These studies eliminate a bottleneck in macrophage production and enable these cells to be engineered and manufactured in a standardized, off-the-shelf manner, rather than on a patient-specific basis. Major Proposed Activities […]